Monday, 12 October 2015

Praising Arizona - Looking back at NACFC 2015

The past four days saw Ed Owen, Trust Chief Executive, lead our delegation to the North American CF Conference in Phoenix, Arizona. Here he gives us the highlights from the largest global gathering in the fight against cystic fibrosis.

As the 4,000 clinicians, health professionals, scientists,
company reps, families and charities like ourselves pack up and head homeward
across the globe, it’s worth reflecting what the 29th annual North American CF Conference has told us about the state of play in our international
effort to beat cystic fibrosis.

Rebecca Cosgriff, Registry Lead, presenting at NACFC

The collective determination and purpose remains very strong
and was evident throughout the four days of meetings, workshops, speeches and
discussions. If anything, the sheer excitement and wonder generated by the
scientific breakthroughs of just a few years ago has given way to a cool
confidence at what can be achieved across a range of fronts.

This is matched, of course, with a realistic view that there
is a long way still to travel before our job is done. But distant hope has
genuinely shifted to steely belief and this conference showed how we, as an
international cystic fibrosis community, are transforming the character and face
of this condition.

A good illustration of this is to witness the attendance of
the multitude of pharmaceutical and biotech companies here in Phoenix. Barely a
decade ago, much of industry would have avoided investing in a ‘rare disease’
like cystic fibrosis. Today, you can’t move for industry talking about their
development of new therapies, devices and diagnostic tools targeted at those
with the condition.

All this is good news but not without its challenges. For
example, Vertex announced
this week further news on its pipeline of precision medicines with planned
trials of new compounds to develop ‘triple therapies’ aimed at correcting the
genetic defect of those with one or two copies of the F508delta mutation.

But, of course, we in the UK are soon to confront the more
immediate challenge of ensuring Orkambi – a mere double therapy of ivacaftor
and lumacaftor – is made available to those who can benefit.

A key figure in the development of the pipeline of
gene-modifying therapies now coming through has been the Cystic Fibrosis
Foundation (CFF)’s long-time President, Bob Beall. After 35 years in his post,
he has made way for his deputy, Preston Campbell, and this conference was the
opportunity for the cystic fibrosis community across the US and worldwide to
mark his extraordinary legacy.

But, as I said, our work is very far from done, and while we
were here we sat down with Preston and his senior team to discuss a range of
collaborations with the Foundation, including the prospect of longer-term
innovation to develop a fundamental cure for cystic fibrosis using stem cell
and gene editing techniques.

And alongside this transformational activity there is the
needs of people with cystic fibrosis community today, and it was refreshing to
hear Preston commit the CFF to a programme of activities to better engage those
with the condition, to hear the diversity of views and experience of the people
we are here for.

As I said in my speech to the UK CF conference in Manchester
last month, cystic fibrosis for those living with it is not primarily a
clinical or scientific issue, it’s a life issue – and we need to understand
that in everything we do to help remove the barriers that prevent people with
CF living the life they want.

Fighting for a life unlimited by cystic fibrosis, today and
tomorrow, is what we are about, and I am pleased to report from the desert
landscape of Arizona that this fight is alive, strong and growing.